Four new drugs for rare diseases on anvil; 5 medications for 7 ailments in mkt: VK Paul

New Delhi, Aug 20 (PTI) Four new drugs for rare diseases are in the pipeline expected to be launched soon while five medications for seven illnesses have already been introduced in the market alleviating suffering of patients, Niti Aayog member (health) Dr VK Paul said on Wednesday.

Speaking at an event here, he noted that generic medications launched by the local companies have led to immense savings for the people suffering from rare diseases.

Currently, 63 rare diseases are included under National Policy for Rare Diseases on recommendation of Central Technical Committee for Rare Diseases (CTCRD).

"For seven diseases, we have made significant progress. These seven diseases include Thalassemia, Wilson's disease and Cystic Fibrosis... there are five drugs which are now available in the market," Paul stated.

Four other drugs are on the anvil, he added.

He noted that NITI Aayog had shortlisted 13 disorders as a priority in 2023.

Paul stated that rare diseases are estimated to collectively affect close to nine crore Indians, making them both a health and societal challenge. He emphasised that with only a fraction of the 7,000 rare diseases having approved therapies, indigenous manufacturing, stronger registries, and multi-ministerial coordination must be prioritised.

He underlined that collective resolve must ensure faster diagnosis, equitable treatment, and an improved quality of life.

Paul also highlighted the importance of collaboration across academia, industry, and government so that quality and affordable treatments can be developed and research can be translated into lasting cures.

Department of Pharmaceuticals Secretary Amit Agrawal said the government under the PLI scheme for pharmaceuticals has been able to support production of drugs to treat eight rare diseases.

He noted that the government aims to treat rare diseases and orphan drugs as a strategic priority area among the priority areas with higher policy support.

Novartis MD & Country President Amitabh Dube said rare diseases affect over 300 million globally, with 70-90 million in India.

Acknowledging the progress made, he stressed the need for continued efforts to address the challenges faced by patients in the rare disease management.

"We have to create an ecosystem that encourages and enables R&D, reduces regulatory delays and values innovation so treatments can reach Indian patients faster," he added.

He also advocated sustainable financing solutions to ensure access to emerging, advanced therapies that exceed Rs 50 lakh. PTI MSS TRB